Results from C91 “Late-Breaking Clinical Trials” on Tuesday afternoon, May 17, will bring new light to clinical problems and potential treatments. The results of one study on the efficacy and safety of sirolimus in lymphangioleiomyomatosis (LAM) were published April 28, 2011, in the The New England Journal of Medicine. While four others examine possible new therapies for people with asthma (pregnant women), emphysema, TB and chronic obstructive pulmonary disease (COPD), a sixth looks at the safety of physician- versus nurse-led transport teams for critically ill patients.

International LAM & Safety Of Sirolimus (Miles) Trial 
LAM is a rare, cystic lung disease of women, caused by mutations in tuberous sclerosis complex (TSC) genes that regulate mammalian target of rapamycin (mTOR) signaling. Sirolimus, which suppresses mTOR activation that occurs when TSC genes are corrupted, has shown promise for patients with TSC or LAM in pilot trials. Researchers conducted a 24-month, randomized, double-blind trial at 13 NIH Rare Lung Disease Consortium sites to determine if sirolimus improves lung function in patients with LAM.

Francis X. McCormack, MD, of the University of Cincinnati School of Medicine

Asthma Exacerbations During Pregnancy Are Reduced By Inflammometry (Feno)-Guided Asthma Management 
Asthma exacerbations during pregnancy are common and associated with significant maternal and fetal morbidity. Inflammometry using induced sputum reduces exacerbations in non-pregnant asthmatic adults, but results from FENO-guided management are equivocal. This double-blind, parallel group, randomized controlled trial enrolled 242 pregnant asthmatic women before 20 weeks gestation. The purpose of this study was to test a new management algorithm for asthma in pregnancy based on FENO and symptoms and compare this to guideline based care.

 Heather Powell, MMedSci, of the Hunter Medical Research Institute at John Hunter Hospital in Newcastle, Australia

Tesra (Treatment Of Emphysema With A Selective Retinoid Agonist) Study 
Palovarotene is an oral gamma-selective retinoid agonist that reduces inflammation and promotes structural repair and functional improvement in animal models of emphysema. TESRA was a Phase II, double-blind, placebo (PL)-controlled randomized, multicenter study assessing the safety and efficacy of 5 mg/day palovarotene given for two years to 492 patients with smoke-induced emphysema established by lung computed tomography and fewer than two COPD exacerbations during the previous year. All patients were treated with tiotropium and a combination of ICS/LABA throughout the study.

Paul W. Jones, MD, PhD, of St. George’s, University of London

A Phase Ii Study Of A Rifapentine-Containing Regimen For Intensive Phase Treatment Of Pulmonary Tb:
Preliminary Results For Tb Trials Consortium Study 29 
Rifapentine administered five days/week has potent activity in a mouse model of anti-tuberculosis (TB) chemotherapy, but data on efficacy and safety in humans are limited. In this study, researchers compared the antimicrobial activity and safety of rifapentine versus rifampin administered five days/week during the first eight weeks of combination therapy for pulmonary TB.

Susan Dorman, MD, of Johns Hopkins University in Baltimore

Chronic Azithromycin Decreases Frequency Of Copd Exacerbations 
Acute exacerbations of chronic obstructive pulmonary disease (AECOPDs) increase the morbidity, mortality and cost of care of patients with COPD. Macrolide antibiotics have antimicrobial and immunomodulatory effects and, when taken chronically, are efficacious in patients with cystic fibrosis and a variety of other inflammatory airways diseases. Seven small studies evaluating the effect of macrolides on AECOPDs report conflicting results. Researchers performed a prospective, randomized, double-blind, placebo-controlled trial to determine if azithromycin decreased AECOPDs.

Richard Albert, MD, of Denver Health Medical Center

Intensive Care Physician- Versus Qualified Nurse- Based Critical Care Transport—
A Randomized Controlled (Iq-Transport Trial) 
The continual regionalization of care increases the need for interhospital critical care transport. The level of expertise of the escorting team to safely execute ground critical care transport is unknown. The use of a digital patient record system during transport, storing all patient monitoring and mechanical ventilation data, enables off-line analysis of critical events during transport possibly unnoticed with manual documentation. This prospective, randomized (stratified for distance), open-label, blinded-endpoint, non-inferiority trial looked at critical care patients transported by the mobile intensive care unit in the Amsterdam region with exclusion criteria. The aim was to evaluate possible risks of interhospital ground critical care transports executed solely by a registered critical care nurse, compared to a combination of nurse and critical care physician, both assisted by a paramedic.

Erik van Lieshout, MD, of the Academic Medical Center at the University of Amsterdam, the Netherlands